Pfiz­er and Sang­amo have dosed their first pa­tient in the Phase III gene ther­a­py tri­al of giroc­toco­gene fitel­par­vovec (SB-525) for he­mo­phil­ia A. Re­searchers will be fol­low­ing the ex­pe­ri­ences of pa­tients for 12 months af­ter dose, check­ing on safe­ty, ef­fi­ca­cy and dura­bil­i­ty. The move comes on the heels of a ma­jor set­back at ri­val Bio­Marin, which re­ceived a re­jec­tion let­ter from the FDA, which de­mand­ed more proof of dura­bil­i­ty — a sore point among some of the an­a­lysts track­ing the pro­gram.

A spokesper­son for Pfiz­er tells End­points News to­day that they plan “a piv­otal read­out in ear­ly 2022 for giroc­toco­gene fitel­par­vovec.”

Bren­da Coop­er­stone, Pfiz­er’s chief de­vel­op­ment of­fi­cer, rare dis­ease, not­ed: “En­roll­ment in the lead-in study is pro­gress­ing well and re­cruit­ment is on track for Phase 3. Giv­en the Phase 1/2 study find­ings to date, we be­lieve that giroc­toco­gene fitel­par­vovec has the po­ten­tial to sus­tain fac­tor lev­els and re­duce an­nu­al bleed rates, sug­gest­ing this one-time gene ther­a­py could po­ten­tial­ly trans­form the stan­dard of care for el­i­gi­ble pa­tients world­wide.” — John Car­roll

Co­di­ak scales down IPO ex­pec­ta­tions to $83M

Amid a siz­zling hot biotech IPO en­vi­ron­ment where most com­pa­nies are bring­ing in more than they ini­tial­ly planned, Co­di­ak Bio­Sciences is tun­ing down ex­pec­ta­tions as it sets terms.

Hav­ing pen­ciled in $100 mil­lion in their IPO fil­ing, the ex­o­some spe­cial­ists now say they plan to raise $83 mil­lion by of­fer­ing 5.5 mil­lion shares at a price range of $14 to $16. That’s low­er than what they would have raised in 2019, when they launched but ul­ti­mate­ly pulled an at­tempt to go pub­lic due to un­fa­vor­able mar­ket con­di­tions.

At the mid­point of the pro­posed range, Co­di­ak would com­mand a ful­ly di­lut­ed mar­ket val­ue of $309 mil­lion, ac­cord­ing to Re­nais­sance Cap­i­tal.

A pi­o­neer in co-opt­ing ex­o­somes, the body’s nat­ur­al in­ter­cel­lu­lar com­mu­ni­ca­tion sys­tem, to de­liv­er ther­a­peu­tic pay­loads to spe­cif­ic ad­dress­es, Co­di­ak’s lead pro­gram is de­signed to ac­ti­vate the STING path­way to treat sol­id tu­mors. It ex­pects to start Phase I/II lat­er this year and reap pre­lim­i­nary da­ta by mid-2021. — Am­ber Tong

Pur­due re­port­ed­ly near­ing guilty plea in fed­er­al opi­oid probe

Pur­due Phar­ma is near­ing a plea deal where it would plead guilty to crim­i­nal charges re­lat­ed to its role in ag­gres­sive­ly mar­ket­ing high­ly ad­dic­tive opi­oids, ac­cord­ing to a Reuters re­port.

Run by the Sack­ler fam­i­ly, Pur­due would re­port­ed­ly face fines ex­ceed­ing $8 bil­lion should the agree­ment be reached. Those fines in­clude about $3.54 bil­lion in a crim­i­nal fine, a $2 bil­lion crim­i­nal for­fei­ture and a $2.8 bil­lion civ­il penal­ty, per Reuters. 

The agree­ment could be reached some­time with­in the next two weeks. Be­cause of Pur­due’s bank­rupt­cy pro­ceed­ings, the fines are un­like­ly to be paid out any­time soon giv­en that the com­pa­ny lacks the cash at present to re­pay all cred­i­tors.

Speak­ing to Reuters, a Jus­tice De­part­ment spokesper­son de­clined to com­ment but said that the re­port “con­tains in­ac­cu­ra­cies and is high­ly mis­lead­ing” with­out ex­plain­ing.

Pros­e­cu­tors across the coun­try, at the fed­er­al and state lev­els, have al­leged Pur­due made bil­lions of dol­lars in prof­its from its opi­oids and sent il­le­gal kick­backs to doc­tors and phar­ma­cies. Pur­due filed for bank­rupt­cy last year fol­low­ing sev­er­al law­suits and crim­i­nal cas­es over its role in fu­el­ing the opi­oid cri­sis. — Max Gel­man

Abeona forms spe­cial com­mit­tee to over­see lead­er­ship

Fol­low­ing a rough few years, Abeona Ther­a­peu­tics an­nounced the for­ma­tion of a spe­cial com­mit­tee of board mem­bers to over­see its ex­ec­u­tive lead­er­ship.

In ad­di­tion, the com­pa­ny has brought in Jef­feries to con­duct a re­view of strate­gic op­er­a­tions. The com­pa­ny said there’s no de­fined time­line for the re­view, and that it doesn’t in­tend to com­ment fur­ther un­less “a spe­cif­ic ini­tia­tive is ap­proved by the board of di­rec­tors, the re­view process is con­clud­ed, or it is oth­er­wise de­ter­mined that oth­er dis­clo­sure is ap­pro­pri­ate.”

Back in 2018, Abeona chief Carsten Thiel was oust­ed fol­low­ing ac­cu­sa­tions of un­spec­i­fied “per­son­al mis­con­duct.” And the fol­low­ing year, the FDA pumped the brakes on the biotech’s Phase III study of its po­ten­tial treat­ment for re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa. The FDA lift­ed its hold in De­cem­ber, al­low­ing Abeona to re­sume test­ing.

Last Oc­to­ber, Abeona’s $ABEO shares were at $2.38. On Tues­day, they closed at $1.22.

The spe­cial com­mit­tee will work “close­ly with the ex­ec­u­tive lead­er­ship team to de­vel­op the com­pa­ny’s strate­gic di­rec­tion and lead­er­ship plan,” Abeona an­nounced. — Nicole De­Feud­is 

Urovant and Sunovion strike mar­ket­ing deal for po­ten­tial over­ac­tive blad­der drug

Urovant has struck a 5-year deal with Sunovion Phar­ma­ceu­ti­cals to mar­ket its over­ac­tive blad­der drug vibegron in pri­ma­ry care in the US, should it get ap­proval in De­cem­ber.

Walt John­ston

The FDA ac­cept­ed Urovant’s NDA for vibegron in March, and set a PDU­FA date for Dec 26. Urovant in-li­censed the drug from Mer­ck with a $25 mil­lion up­front. Topline Phase III da­ta showed that it hit sta­tis­ti­cal sig­nif­i­cance for both co-pri­ma­ry end­points and all 7 sec­on­daries when com­pared against a place­bo.

Ac­cord­ing to the com­pa­ny, it’s al­so be­ing eval­u­at­ed for OAB in men with be­nign pro­sta­t­ic hy­per­pla­sia, and for ab­dom­i­nal pain as­so­ci­at­ed with ir­ri­ta­ble bow­el syn­drome.

“While Urovant sales rep­re­sen­ta­tives will be fo­cused on urol­o­gists, long-term care, and high-pre­scrib­ing PCPs, the Sunovion team will sig­nif­i­cant­ly broad­en our reach in­to the U.S. pri­ma­ry care com­mu­ni­ty,” Urovant’s se­nior com­mer­cial VP Walt John­ston said in a state­ment.

The new deal is in ad­di­tion to an ex­clu­sive 3-year agree­ment the com­pa­nies reached in June for whole­sale trade and re­tail dis­tri­b­u­tion, con­tract op­er­a­tions and se­lect ac­count man­age­ment for vibegron. — Nicole De­Feud­is 

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.